Completed

NTBC Enzyme Inhibitor Treatment for Tyrosinemia Type I

0 criteria met from your profileSee at a glance how your profile meets each eligibility criteria.
What is being tested

NTBC

Drug
Who is being recruted

Amino Acid Metabolism, Inborn Errors+13

+ Brain Diseases

+ Brain Diseases, Metabolic

See all eligibility criteria
How is the trial designed

Treatment Study

Phase 2
Interventional
Study Start: November 1994
See protocol details

Summary

Principal SponsorNational Center for Research Resources (NCRR)
Last updated: June 24, 2005
Sourced from a government-validated database.Claim as a partner

Study start date: November 1, 1994

Actual date on which the first participant was enrolled.

PROTOCOL OUTLINE: Only 2 patients with tyrosinemia type I are known to this research team; others will be treated if found and if clinical conditions permit. The enzyme inhibitor 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione (NTBC) is administered orally, in 2 divided doses daily with meals. Patients will be followed closely for side effects attributable to NTBC.

NCT00004333
Principal SponsorNational Center for Research Resources (NCRR)
Last updated: June 24, 2005
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Amino Acid Metabolism, Inborn ErrorsBrain DiseasesBrain Diseases, MetabolicCentral Nervous System DiseasesMetabolic DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNutritional and Metabolic DiseasesPathologic ProcessesPathological Conditions, Signs and SymptomsBrain Diseases, Metabolic, InbornDisease AttributesGenetic Diseases, InbornMetabolism, Inborn ErrorsTyrosinemiasRare Diseases

Criteria

PROTOCOL ENTRY CRITERIA: Tyrosinemia type I No cirrhosis No hepatocellular carcinoma

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.
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CompletedNo study centers